The evolving value assessment of cancer therapies: Principles from the cancer community.
Authors: Myrto Lee, Anne-Marie Baird, Susan Brown, Russell Clark, Javier Cortes, Giuseppe Curigliano, Andrea Stern Ferris, Ataru Igarashi, Ravindran Kanesvaran, Hugo Larose, Gary Lyman, Luca Pani, Zack Pemberton-Whiteley, Tomas Salmonson, Peter Sawicki, Barry D. Stein, Dong-Churl Suh, Galina Velikova, Richard Vines, Jens Grueger
Journal: Journal of Clinical Oncology (2023)

e13647 Background: Oncology-relevant measures of disease and treatment support earlier detection, accelerated drug development, and inform treatment pathways. To advance access to transformative treatments for people with cancer, especially those for early-stage cancers where medical intervention has the greatest opportunity to impact the course of the disease, the cancer community identified seven principles that support the evolution of value assessment frameworks. Here we summarize the five key principles. Methods: A group of 24 cancer community experts (9 oncologists, 6 patient advocacy leaders, 1 former regulator, 6 former payers, and 4 health economists) from 14 countries came together to discuss and build consensus on oncology-relevant value principles. Over five months, the expert group engaged via surveys, virtual discussion panels, and interviews. Results: The expert group developed seven consensus principles covering two areas: oncology-relevant endpoints to assess the value of treatments; and additional important value components and how they can be integrated into value assessments. The key 5 principles were oncology-relevant endpoints assessing clinical effectiveness, including the following three items. 1) Consider oncology-relevant endpoints, other than Overall Survival (OS), with intrinsic value for decision-making e.g., Event-, Disease-, or Relapse-Free Survival. 2) Continue to build evidence for endpoints that indicate treatment efficacy earlier e.g., pathologic Complete Response. 3) Develop evidence for the next generation of predictive measures that detect and monitor disease, e.g., circulating tumor DNA and Minimal Residual Disease. Additional value components supporting patient access include the following two items. 1) Use managed entry (type of access) agreements supported by ongoing real world evidence collection to help address decision-maker (regulator and Health Technology Assessment body) evidence needs. 2) Routinely use Patient Reported Outcomes (PROs) in value assessments. Conclusions: To achieve the common goal of all cancer community stakeholders—improved outcomes for people with cancer—collaboration across the cancer community is essential to define how value is assessed, especially for early-stage cancer treatments. This is particularly important since mature OS data is not always available during initial decision-making, and decision-makers need to evolve which are the most appropriate endpoints. This supports measuring clinical impact, but also broader social, financial and ethical impact for patients, caregivers, healthcare systems, society, and the economy (not considered in this abstract). To achieve these goals, the expert group recommends: 1) increasing awareness and systematically engaging people with cancer in value assessments, 2) leveraging PROs in those assessments, and 3) further validation in clinical trials of oncology-relevant endpoints that could provide an earlier indication of clinical outcomes.